The Cystic Fibrosis Foundation has surpassed $100 million in research funding through its Infection Research Initiative, created to help address the chronic and intractable infections that are a hallmark of cystic fibrosis. Reflecting the momentum and focus of this initiative, the Foundation hit this milestone in a little over three years, nearly two years ahead of its five-year commitment.

The current funding stands at more than $109 million. The Foundation aims to continue to support infection research at the same pace as it has in the past three years.

“Reaching this goal doesn’t mean we have reached the finish line. We will maintain this momentum and continue to fund any science that we believe holds real promise to address infections,” said Tiffany Burnett, co-lead of the Infection Research Initiative and senior director of biopharma programs at the Cystic Fibrosis Foundation. “Chronic infections are a serious threat to the health of people with CF, and we continue to need improved detection and more effective treatments.”

People with cystic fibrosis who have chronic infections are at greater risk for worsening lung disease, and infection remains a top concern for both patients and clinicians. In 2018, the CF Foundation dedicated at least $100 million to its Infection Research Initiative, a comprehensive approach to improve outcomes associated with infections through enhanced detection, diagnosis, prevention, and treatment.

The Foundation’s investment in this initiative paves the way for our continuing efforts to pursue novel and non-traditional ways to treat drug-resistant infections, optimize current therapies, and expand our work into better detection and diagnosis. A major focus moving forward is the development of new technologies for quicker, more accurate tests to detect and diagnose infections beyond sputum.

Our Infection Research

Since the start of the Infection Research Initiative, the Foundation has funded more than 20 industry programs focused on developing new treatments and more than 200 academic research projects. The majority of the funding -- $60 million -- has gone to developing new treatments. The initiative ushered in a dramatic uptick in the number of Foundation-supported studies with a 67% average annual increase in the number of studies compared to 2018, the year before the start of the initiative. 

Industry Highlights

Many people with CF live in fear of contracting an infection for which existing antibiotics are not effective. To address the increasing problem of antibiotic resistance, the Foundation is supporting the development of new treatments to tackle difficult-to-treat bacteria. We now have six studies investigating treatments for Pseudomonas (and other disease-causing germs), five studies for nontuberculous mycobacteria (NTM), and two studies for methicillin-resistant Staphylococcus aureus (MRSA). Non-traditional ways to address these and other difficult-to-treat bacteria are also being explored, including the use of nitric oxide, IV gallium, and bacteriophage (phage) therapies. 

Nitric oxide, a gas molecule produced by the body, plays a key role in the immune system. Researchers believe that increasing levels of nitric oxide in the body could help eliminate bacteria and increase lung function in people with CF. Gallium, a molecule nearly identical to iron that disrupts iron-dependent biological processes of bacteria, has already been approved for IV use for people with other illnesses. IV gallium is being studied for its effectiveness in treating infections in people with CF. The Foundation is investing in several programs testing phage therapy, the use of phages to treat an infection in a person. Phages are specialized viruses that kill very specific bacterial strains. 

Academic Highlights

The Foundation has funded cutting-edge infection research at institutions around the country. To begin to tackle better detection and diagnosis of infections, the Foundation has committed close to $6 million to help identify emerging technologies in the field of detection that do not require sputum. The reason for this effort is that it is becoming more difficult to test for infections as people with CF who are taking Trikafta^® are less able to produce sputum on demand or have significant decreases in the amount of sputum they can produce. Providing alternative diagnostic tests to inform care and advance drug development and clinical research is vital moving forward. 

Marking a highlight of our ongoing academic research, the Foundation recently received the results to the STOP2 clinical trial that showed that a longer course of IV antibiotics was not more effective than a shorter course in treating a pulmonary exacerbation in either rapid or slower responders. Ultimately, this study will help reduce the time people with CF spend on IV antibiotics, hopefully leading to fewer side effects and a lower risk of antibiotic resistance.

In continuation of the STOP studies, the STOP-PEDS pilot study is underway. This study will evaluate children 6 to 18 years old with CF to determine the acceptability and feasibility of a larger trial, comparing whether to treat mild pulmonary exacerbations with antibiotics right away versus waiting to see whether increased airway clearance or another approach is enough to make symptoms better. 

The Foundation also is supporting the development of STOP3. The first study of the STOP3 program will evaluate if the addition of an IV aminoglycoside to IV beta lactam antibiotic treatment is superior to just a beta lactam antibiotic to treat Pseudomonas. This study hopes to show that two IV drugs (one of which is an aminoglycoside) isn’t superior to one IV drug in treating an exacerbation. This is important because aminoglycosides can negatively affect hearing and kidney function. This toxicity must come with clear benefit to justify continued use. 

A Look Ahead

The Foundation will continue to fund infection research to address the unmet needs of people with CF. One example of this is our upcoming effort to design a large-scale study to treat Aspergillus infections in people with CF. The study, which is expected to start in 2023, is intended to help us better understand fungal infections in people with CF and create the infrastructure needed to test future therapies and detection methods. Pilot studies to support the large-scale study are currently underway. The Foundation also is funding academic research focused on optimizing current therapies and understanding the future of infections in the era of modulators.

Our infection research also has the potential to benefit the broader public. Three of the seven most common infections in people with CF -- caused by Aspergillus, MRSA, and Pseudomonas -- are also serious threats to the general population. 

To ensure that new infection treatments that stem from our infection research make it into the hands of patients, we will continue to promote a sustainable and robust antibiotics pipeline and a marketplace that rewards innovation. As part of this effort, we will continue to advocate for the PASTEUR Act -- bipartisan federal legislation that, if passed, will support the development of new antibiotics and promote appropriate use of existing ones.